Drug Repurposing

Our initial focus was on supporting drug repurposing research using animal models of MEPAN. Repurposing projects can test thousands of FDA-approved drugs to identify existing medicines that may address MEPAN symptoms.

 

Because these drugs have already been reviewed by the FDA and tested in patients for safety, repurposing is potentially the fastest, most cost-effective path to finding a treatment. MEPAN Foundation has funded:

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• A Perlara project to test repurposed drugs in fibroblasts from MEPAN patients. Several of these are were derivative from a 2023 paper from the Bellen Lab at Baylor University that shows iron dysregulation may underly disease pathology in MEPAN and may be helped by reducing levels of ceramides in patients.

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• A pilot project at Kenyon College that tested drug combinations in roundworms with mecr-1 deficiency that provided insight on certain drugs that could well together as part of a multi-drug treatment strategy for MEPAN.

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